scholarly journals Long-Term Hematopoietic Stem Cell Damage in a Murine Model of the Hematopoietic Syndrome of the Acute Radiation Syndrome

2012 ◽  
Vol 103 (4) ◽  
pp. 356-366 ◽  
Author(s):  
Hui Lin Chua ◽  
P. Artur Plett ◽  
Carol H. Sampson ◽  
Mandar Joshi ◽  
Rebeka Tabbey ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell ◽  
Murine Model ◽  
Cell Damage ◽  
Acute Radiation ◽  
Hematopoietic Stem ◽  
Acute Radiation Syndrome

Long-term efficacy and safety of hematopoietic stem cell gene therapy mediated by lentiviral vectors in the murine model of Wiskott-Aldrich syndrome

2008 ◽  
Vol 40 (2) ◽  
pp. 256-257
Author(s):  
Marita Bosticardo ◽  
Francesco Marangoni ◽  
Michela Locci ◽  
Elena Draghici ◽  
Samantha Scaramuzza ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Hematopoietic Stem Cell ◽  
Murine Model ◽  
Efficacy And Safety ◽  
Hematopoietic Stem ◽  
Cell Gene ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy

Long-term hematopoietic stem cell gene therapy corrects neuromuscular manifestations in preclinical study of Pompe mice

2021 ◽  
Vol 132 (2) ◽  
pp. S107
Author(s):  
Niek P. van Til ◽  
Yildirim Dogan ◽  
Cecilia Barese ◽  
Zeenath Unnisa ◽  
Swaroopa Guda ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Hematopoietic Stem Cell ◽  
Preclinical Study ◽  
Hematopoietic Stem ◽  
Cell Gene ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy

scholarly journals Clinical and immunologic outcome of patients with cartilage hair hypoplasia after hematopoietic stem cell transplantation

Blood ◽  
2010 ◽  
Vol 116 (1) ◽  
pp. 27-35 ◽  
Author(s):  
Victoria Bordon ◽  
Andrew R. Gennery ◽  
Mary A. Slatter ◽  
Els Vandecruys ◽  
Genevieve Laureys ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Clinical Manifestations ◽  
Hematopoietic Stem ◽  
Cartilage Hair Hypoplasia ◽  
Severe Immunodeficiency

Abstract Cartilage-hair hypoplasia (CHH) is a rare autosomal recessive disease caused by mutations in the RMRP gene. Beside dwarfism, CHH has a wide spectrum of clinical manifestations including variable grades of combined immunodeficiency, autoimmune complications, and malignancies. Previous reports in single CHH patients with significant immunodeficiencies have demonstrated that allogeneic hematopoietic stem cell transplantation (HSCT) is an effective treatment for the severe immunodeficiency, while growth failure remains unaffected. Because long-term experience in larger cohorts of CHH patients after HSCT is currently unreported, we performed a European collaborative survey reporting on 16 patients with CHH and immunodeficiency who underwent HSCT. Immune dysregulation, lymphoid malignancy, and autoimmunity were important features in this cohort. Thirteen patients were transplanted in early childhood (∼ 2.5 years). The other 3 patients were transplanted at adolescent age. Of 16 patients, 10 (62.5%) were long-term survivors, with a median follow-up of 7 years. T-lymphocyte numbers and function have normalized, and autoimmunity has resolved in all survivors. HSCT should be considered in CHH patients with severe immunodeficiency/autoimmunity, before the development of severe infections, major organ damage, or malignancy might jeopardize the outcome of HSCT and the quality of life in these patients.

scholarly journals Long-term outcome following hematopoietic stem-cell transplantation in Wiskott-Aldrich syndrome: collaborative study of the European Society for Immunodeficiencies and European Group for Blood and Marrow Transplantation

Blood ◽  
2008 ◽  
Vol 111 (1) ◽  
pp. 439-445 ◽  
Author(s):  
Hulya Ozsahin ◽  
Marina Cavazzana-Calvo ◽  
Luigi D. Notarangelo ◽  
Ansgar Schulz ◽  
Adrian J. Thrasher ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Collaborative Study ◽  
Hematopoietic Stem ◽  
Long Term Outcome ◽  
Wiskott Aldrich Syndrome

Wiskott-Aldrich syndrome (WAS) is a rare X-linked immunodeficiency with microthrombocytopenia, eczema, recurrent infections, autoimmune disorders, and malignancies that are life-threatening in the majority of patients. In this long-term, retrospective, multicenter study, we analyzed events that occurred in 96 WAS patients who received transplants between 1979 and 2001 who survived at least 2 years following hematopoietic stem-cell transplantation (HSCT). Events included chronic graft-versus-host disease (cGVHD), autoimmunity, infections, and sequelae of before or after HSCT complications. Three patients (3%) died 2.1 to 21 years following HSCT. Overall 7-year event-free survival rate was 75%. It was lower in recipients of mismatched related donors, also in relation with an older age at HSCT and disease severity. The most striking finding was the observation of cGVHD-independent autoimmunity in 20% of patients strongly associated with a mixed/split chimerism status (P < .001), suggesting that residual-host lymphocytes can mediate autoimmune disease despite the coexistence of donor lymphocytes. Infectious complications (6%) related to splenectomy were also significant and may warrant a more restrictive approach to performing splenectomy in WAS patients. Overall, this study provides the basis for a prospective, standardized, and more in-depth detailed analysis of chimerism and events in long-term follow-up of WAS patients who receive transplants to design better-adapted therapeutic strategies.

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